WO2021247856A3 - Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems - Google Patents

Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems Download PDF

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Publication number
WO2021247856A3
WO2021247856A3 PCT/US2021/035691 US2021035691W WO2021247856A3 WO 2021247856 A3 WO2021247856 A3 WO 2021247856A3 US 2021035691 W US2021035691 W US 2021035691W WO 2021247856 A3 WO2021247856 A3 WO 2021247856A3
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methods
lineage
crispr
inhibition
compositions
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PCT/US2021/035691
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French (fr)
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WO2021247856A2 (en
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Siddhartha MUKHERJEE
Florence Borot
Abdullah Mahmood Ali
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The Trustees Of Columbia University In The City Of New York
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Priority to JP2022574196A priority Critical patent/JP2023528415A/en
Priority to EP21817226.0A priority patent/EP4161535A2/en
Priority to MX2022015237A priority patent/MX2022015237A/en
Priority to CA3180738A priority patent/CA3180738A1/en
Priority to US17/928,674 priority patent/US20240041923A1/en
Priority to KR1020227043770A priority patent/KR20230029632A/en
Priority to AU2021285907A priority patent/AU2021285907A1/en
Priority to CN202180060702.7A priority patent/CN116157135A/en
Publication of WO2021247856A2 publication Critical patent/WO2021247856A2/en
Publication of WO2021247856A3 publication Critical patent/WO2021247856A3/en

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Abstract

Disclosed herein are methods of administering an agent targeting a lineage- specific cell-surface antigen, e.g., CD33 or EMR2, and a population of hematopoietic cells that are altered in the expression of the lineage- specific cell- surface antigen, e.g., CD33 or EMR2, for immunotherapy of hematological malignancies. Also disclosed herein are methods of administering an agent targeting more than one lineage-specific cell- surface antigen, and a population of hematopoietic cells that are altered in the expression of more than one lineage- specific cell-surface antigen, for immunotherapy of hematological malignancies. Cells comprising mutations in CD33, or EMR2, or more than one lineage- specific cell-surface antigen are also provided, as are methods of producing such cells using CRISPR-based base editor systems.
PCT/US2021/035691 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems WO2021247856A2 (en)

Priority Applications (8)

Application Number Priority Date Filing Date Title
JP2022574196A JP2023528415A (en) 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage-specific antigens using CRISPR-based base editor systems
EP21817226.0A EP4161535A2 (en) 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems
MX2022015237A MX2022015237A (en) 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems.
CA3180738A CA3180738A1 (en) 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems
US17/928,674 US20240041923A1 (en) 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems
KR1020227043770A KR20230029632A (en) 2020-06-03 2021-06-03 Compositions and methods for inhibiting lineage specific antigens using a CRISPR-based base editor system
AU2021285907A AU2021285907A1 (en) 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems
CN202180060702.7A CN116157135A (en) 2020-06-03 2021-06-03 Compositions and methods for inhibiting lineage specific antigens using CRISPR-based base editor systems

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202063033966P 2020-06-03 2020-06-03
US202063033970P 2020-06-03 2020-06-03
US63/033,966 2020-06-03
US63/033,970 2020-06-03
US202163183791P 2021-05-04 2021-05-04
US63/183,791 2021-05-04

Publications (2)

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WO2021247856A2 WO2021247856A2 (en) 2021-12-09
WO2021247856A3 true WO2021247856A3 (en) 2022-01-13

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PCT/US2021/035691 WO2021247856A2 (en) 2020-06-03 2021-06-03 Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems

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US (1) US20240041923A1 (en)
EP (1) EP4161535A2 (en)
JP (1) JP2023528415A (en)
KR (1) KR20230029632A (en)
CN (1) CN116157135A (en)
AU (1) AU2021285907A1 (en)
CA (1) CA3180738A1 (en)
MX (1) MX2022015237A (en)
WO (1) WO2021247856A2 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4048790A4 (en) * 2019-10-22 2024-03-27 Fred Hutchinson Cancer Center Base editor-mediated cd33 reduction to selectively protect therapeutic cells
CA3236152A1 (en) * 2021-11-09 2023-05-19 Vor Biopharma Inc. Compositions and methods for erm2 modification
WO2024073606A1 (en) * 2022-09-28 2024-04-04 Regeneron Pharmaceuticals, Inc. Antibody resistant modified receptors to enhance cell-based therapies

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018232039A1 (en) * 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Methods for modifying rna splicing
WO2019046285A1 (en) * 2017-08-28 2019-03-07 The Trustees Of Columbia University In The City Of New York Cd33 exon 2 deficient donor stem cells for use with cd33 targeting agents

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018232039A1 (en) * 2017-06-14 2018-12-20 Ptc Therapeutics, Inc. Methods for modifying rna splicing
WO2019046285A1 (en) * 2017-08-28 2019-03-07 The Trustees Of Columbia University In The City Of New York Cd33 exon 2 deficient donor stem cells for use with cd33 targeting agents

Also Published As

Publication number Publication date
US20240041923A1 (en) 2024-02-08
EP4161535A2 (en) 2023-04-12
KR20230029632A (en) 2023-03-03
JP2023528415A (en) 2023-07-04
CN116157135A (en) 2023-05-23
WO2021247856A2 (en) 2021-12-09
MX2022015237A (en) 2023-02-09
CA3180738A1 (en) 2021-12-09
AU2021285907A1 (en) 2023-02-02

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